Research & Development Solution Center

In the life sciences industry, we are always hyper-vigilant about all of the risks involved in product development. Therefore, our “cuttingedge” approaches seem not quite as innovative as other high-tech industries. The time has come for a few key best practices that add efficiency and cost-effectiveness, something the clinical development world desperately needs. In order to confirm my theories, I queried a number of executivelevel professional colleagues in pharmaceutical and biotechnology companies, who shared their views off the record. I analyzed my queries and compiled the results into a cohesive discussion below. To add a note of practicality to each, I’ll suggest considerations for those who want to make it happen in the present and look toward the future.

Since the discovery of penicillin in 1929, the birth of the antimicrobial era meant the beginning of a brave new world, where people no longer died from common bacterial infections, and surgery could be approached with confidence.

Prior to heading out to the 32nd Annual J.P. Morgan Healthcare Conference in San Francisco last month, I stumbled across several article headlines indicating the United States’ domination of global biomedical R&D was fading. What metric was used to determine this? R&D spend. According to “new” research from the University of Michigan Health System, the U.S. share of the global biomedical R&D business declined from 51 percent to a mere 45 percent from 2007 to 2012. And while Europe remained unchanged at 29 percent, Asia rose from 18 to 24 percent.

When it comes to site monitoring, pharmaceutical companies have traditionally taken a conservative approach, performing frequent on-site monitoring and 100 percent verification of all data. This practice goes beyond what’s required, says the FDA in its Guidance for Industry: Oversight of Clinical Investigations — Risk-Based Approach to Monitoring, issued in August 2013. The agency asserts that sponsors adopted current practices based on a “perception” that 100 percent source data verification (SDV) was the FDA’s preferred way to meet monitoring obligations.

During the ill-fated 1970 Apollo 13 mission to the moon, it was astronaut Jack Swigert who alerted ground control that something had gone terribly wrong when he uttered the phrase, “Houston, we’ve had a problem here.” Those same words seem very fitting to the current state of affairs around the skyrocketing costs of drug discovery. Recent estimates place the expense of successfully bringing just one drug to market at between $350 million and $1.2 billion. However, in the last decade, companies having brought 4 to 13 drugs to market have watched the price tag reach stratospheric heights — orbiting $5 billion+. “I think the pain point has reached a threshold that’s no longer bearable,” states Dalvir Gill, Ph.D., CEO of TransCelerate BioPharma.

Ever heard of the KISS (keep it simple, stupid) principle? The general idea behind it is that systems perform best when the design is simple, not complex. My favorite example demonstrating the application of KISS, as well as the impact of failing to do so, is captured in a scene in the 1995 movie Apollo 13. An incident necessitates three astronauts use the lunar module (LM), a ship built just for landing on the moon, as a lifeboat to survive.

The U.S. economy has been unstable for many years now, and this year, the U.S. experienced the first government shutdown since late 1995, causing many investors to become wary of the market. In spite of economic uncertainty over the budget deficit, biotechs remained a “safe haven,” according to CNBC.

According to the Business Perspectives on Emerging Markets 2012-2017 Report by Global Intelligence Alliance (GIA), Brazil, Russia, India and China (BRIC) will retain their leading positions as the worlds’ top emerging markets for 2012-2017. In a ranking of the top 30 emerging markets international companies plan to target in 2012-2017, most of the non-BRIC emerging markets are in Asia or Latin America, with Indonesia, South Africa, Vietnam, Mexico, Turkey and Argentina ranking highest amongst secondary emerging markets.

The jury is still out on the ultimate impact of healthcare reform in general, and perhaps this impact is even murkier in the life sciences space. Certainly there will be very dramatic changes to the providers and payers, but it is more difficult to see exactly how the changes will manifest themselves for the remaining entities in the health sciences arena.

“With revenues growing, it is no surprise that biotech firms are again investing substantial dollars in research and development,” said Ryan Starkes, partner and leader of the Life Sciences Practice at BDO. “However, broader economic challenges persist. Smaller companies are struggling to generate returns on their past investments, and continue to target innovative medicines in hopes of greater revenues down the road.”